- Conditions
- Adrenomyeloneuropathy, Adrenoleukodystrophy
- Interventions
- Lorenzo's oil
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 240 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
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Showing 25–44
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- Conditions
- Adenovirus, Anesthesia, Anxiety, Anxiolysis, Autism, Autistic Disorder, Bacterial Meningitis, Bacterial Septicemia, Benzodiazepine, Bipolar Disorder, Bone and Joint Infections, Central Nervous System Infections, Convulsions, Cytomegalovirus Retinitis, Early-onset Schizophrenia Spectrum Disorders, Epilepsy, General Anesthesia, Gynecologic Infections, Herpes Simplex Virus, Infantile Hemangioma, Infection, Inflammation, Inflammatory Conditions, Intra-abdominal Infections, Lower Respiratory Tract Infections, Migraines, Pain, Pneumonia, Schizophrenia, Sedation, Seizures, Skeletal Muscle Spasms, Skin and Skin-structure Infections, Treatment-resistant Schizophrenia, Urinary Tract Infections, Withdrawal, Sepsis, Gram-negative Infection, Bradycardia, Cardiac Arrest, Cardiac Arrhythmia, Staphylococcal Infections, Nosocomial Pneumonia, Neuromuscular Blockade, Methicillin Resistant Staphylococcus Aureus, Endocarditis, Neutropenia, Headache, Fibrinolytic Bleeding, Pulmonary Arterial Hypertension, CMV Retinitis, Hypertension, Chronic Kidney Diseases, Hyperaldosteronism, Hypokalemia, Heart Failure, Hemophilia, Heavy Menstrual Bleeding, Insomnia
- Interventions
- The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:
- Drug
- Lead sponsor
- Daniel Benjamin
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 3,520 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2019
- U.S. locations
- 43
- States / cities
- Anchorage, Alaska • Little Rock, Arkansas • La Jolla, California + 37 more
- Conditions
- Healthy
- Interventions
- OP-101
- Drug
- Lead sponsor
- Orpheris, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
- Interventions
- hematopoietic stem cell infusion
- Biological
- Lead sponsor
- Talaris Therapeutics Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
- Interventions
- Cholic Acids
- Drug
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1992 – 2009
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Adrenoleukodystrophy
- Interventions
- glyceryl trierucate/glyceryl trioleate
- Drug
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Other
- Eligibility
- 18 Months to 8 Years · Male only
- Enrollment
- 126 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2014
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland
- Conditions
- Adrenoleukodystrophy
- Interventions
- Not listed
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- 1 Year to 5 Years · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
- Interventions
- no intervention
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Not listed
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- X-Linked Adrenoleukodystrophy
- Interventions
- Not listed
- Lead sponsor
- NeuroVia, Inc.
- Industry
- Eligibility
- 2 Years to 13 Years · Male only
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 3
- States / cities
- Palo Alto, California • Boston, Massachusetts • Pittsburgh, Pennsylvania
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
- Interventions
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 135 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1995 – 2010
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Neurodegenerative Diseases, Leukodystrophy, Ataxia, LBSL, Leukoencephalopathy with Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation, Cerebellar Ataxia, Adrenomyeloneuropathy
- Interventions
- Home exercise
- Behavioral
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Other
- Eligibility
- 5 Years to 75 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Obesity, Addison's Disease
- Interventions
- Hydrocortisone, Isocaloric Diet
- Drug · Dietary Supplement
- Lead sponsor
- Oregon Health and Science University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2004
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders
- Interventions
- Campath-1H, Cyclophosphamide, Busulfan, Allogeneic stem cell transplantation, Cyclosporine A, Mycophenolate Mofetil
- Drug · Procedure
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2017
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
- Interventions
- data collection
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 60 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2028
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Adrenoleukodystrophy, Restless Legs Syndrome
- Interventions
- Pramipexole, Placebo
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years to 75 Years · Female only
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Not listed
- Enrollment
- 12,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2030
- U.S. locations
- 23
- States / cities
- Los Angeles, California • Orange, California • Palo Alto, California + 18 more
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts 1, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus-Merzbacher-Like Disease, 1, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Up to 18 Years
- Enrollment
- 236 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2024
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Interventions
- Natural History Observation
- Other
- Lead sponsor
- SwanBio Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 4
- States / cities
- Stanford, California • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Prader Willi Syndrome, Adrenal Insufficiency
- Interventions
- Low dose (1 mcg) ACTH stimulation test, Overnight metyrapone test
- Other
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 2 Years and older
- Enrollment
- 23 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- ALD (Adrenoleukodystrophy), Adrenoleukodystrophy, Cerebral Adrenoleukodystrophy
- Interventions
- Medical Record Abstraction, Biospecimen Sample Collection
- Other
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2030
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota